Key points from article :
Elliott Collins, a 34-year-old from Colchester, has undergone a groundbreaking gene therapy for haemophilia B, a condition he has struggled with for 34 years. Haemophilia B is a bleeding disorder where the body lacks sufficient clotting factor IX, leading to spontaneous and severe bleeding.
Before this treatment, Elliott needed regular injections of factor IX, twice a week, which were crucial to prevent dangerous bleeds and manage his condition.
The gene therapy, known as Hemgenix, is a revolutionary approach that costs £2.6 million and was recently approved for use on the NHS. It works by using engineered viruses to deliver a functional copy of the factor IX gene directly to the liver. This process enables the liver to produce the necessary clotting protein on its own, potentially eliminating the need for regular injections.
Elliott participated in clinical trials starting in 2019 and reports that since the treatment, he has not needed any further factor IX injections. His factor IX levels have risen to 60% of normal, and he describes the therapy as life-changing, giving him a "new lease of life."
The gene therapy is now available at eight NHS centers across the UK, including in Oxford, Manchester, and London. The NHS, in collaboration with CSL Behring and the National Institute for Health and Care Excellence, has negotiated a performance-related payment deal for this treatment.
This means the NHS will pay based on how long the therapy effectively maintains factor IX levels. While the cost is high, it is a significant advancement in treating haemophilia B, and its success could set a precedent for similar therapies for other conditions.
